Antineoplaston Therapy in Treating Children With Primitive Neuroectodermal Tumors (PNET)

• ClinicalTrials.gov Identifier: NCT00003460
• Recruitment Status: Completed
• First Posted: January 27, 2003
• Results First Posted: November 10, 2016
• Last Update Posted: August 24, 2017
• Sponsor: Burzynski Research Institute
• Information provided by (Responsible Party): Burzynski Research Institute

Study Description

Brief Summary:

RATIONALE: Current therapies for children with primitive neuroectodermal tumors that have not responded to standard therapy provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of children with primitive neuroectodermal tumors that have not responded to standard therapy.

PURPOSE: This study is being performed to determine the effects (good and bad) that Antineoplaston therapy has on children (> 6 months of age) with primitive neuroectodermal tumors that has not responded to standard therapy.

Condition or disease	Intervention/treatment	Phase
Childhood CNS Primitive Neuroectodermal Tumor	Drug: Antineoplaston therapy (Atengenal + Astugenal)	Phase 2

Detailed Description:

OBJECTIVES:

• To determine the efficacy of Antineoplaston therapy in children with primitive neuroectodermal tumors that has not responded to standard therapy, as measured by an objective response to therapy (complete response, partial response or stable disease).
• To determine the safety and tolerance of Antineoplaston therapy in children with a brain tumor.

OVERVIEW: This is a single arm, open-label study in which children with primitive neuroectodermal tumors that have not responded to standard therapy receive gradually escalating doses of intravenous Antineoplaston therapy (Atengenal + Astugenal) until the maximum tolerated dose is reached. Treatment continues for at least 12 months in the absence of disease progression or unacceptable toxicity. After 12 months, patients with a complete or partial response or with stable disease may continue treatment.

To determine objective response, tumor size is measured utilizing MRI scans, which are performed every 8 weeks for the first two years, every 3 months for the third and fourth years, every 6 months for the 5th and sixth years, and annually thereafter.

PROJECTED ACCRUAL: A total of 20-40 patients will be accrued to this study.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 13 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Phase II Study of Antineoplastons A10 and AS2-1 In Children With Primitive Neuroectodermal Tumors
• Study Start Date: April 1996
• Actual Primary Completion Date: February 2005
• Actual Study Completion Date: February 2005

Arms and Interventions

Arm	Intervention/treatment
Experimental: Antineoplaston therapy; Antineoplaston therapy (Atengenal + Astugenal) by IV infusion every four hours for at least 12 months. Study subjects receive increasing dosages of Atengenal and Astugenal until the maximum tolerated dose is reached.	Drug: Antineoplaston therapy (Atengenal + Astugenal); Children with a primitive neuroectodermal tumor that has not responded to standard therapy will receive Antineoplaston therapy (Atengenal + Astugenal).; Other Name: A10 (Atengenal); AS2-1 (Astugenal)

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With Objective Response [ Time Frame: 12 months ]
   Objective response rate per Response Assessment in Neuro-Oncology (RANO) for target lesions and assessed by MRI: Complete Response (CR), disappearance of all disease sustained for at least four weeks; Partial Response (PR), >=50% decrease in the sum of the products of of the greatest perpendicular diameters of all measurable enhancing lesions, sustained for at least four weeks.

Secondary Outcome Measures :

1. Percentage of Participants Who Survived [ Time Frame: 6 months, 12 months, 24 months, 36 months, 48 months, 60 months ]
   6 months, 12 months, 24 months, 36 months, 48 months, 60 months overall survival

Eligibility Criteria

• Ages Eligible for Study: 6 Months to 17 Years (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

DISEASE CHARACTERISTICS:

• Histologically confirmed (except if medically contraindicated) incurable primitive neuroectodermal tumor
• Evidence of progressive or recurrent tumor by MRI scan performed within 2 weeks prior to study entry
• Must have received and failed prior standard therapy
• Tumor must be at least 5 mm

PATIENT CHARACTERISTICS:

Age:

• 6 months to 17 years

Performance status:

• Karnofsky 60-100%

Life expectancy:

• At least 2 months

Hematopoietic:

• WBC at least 2000/mm^3
• Platelet count greater than 50,000/mm^3

Hepatic:

• Bilirubin no greater than 2.5 mg/dL
• SGOT and SGPT no greater than 5 times upper limit of normal
• No hepatic insufficiency

Renal:

• Creatinine no greater than 2.5 mg/dL
• No history of renal conditions that contraindicate high dosages of sodium

Cardiovascular:

• No uncontrolled hypertension
• No severe heart disease
• No history of congestive heart failure
• No other cardiovascular conditions that contraindicate high dosages of sodium

Pulmonary:

• No severe lung disease

Other:

• Not pregnant or nursing
• Fertile patients must use effective contraception during and for 4 weeks after study participation
• No serious active infections or fever
• No other serious concomitant disease

PRIOR CONCURRENT THERAPY:

Biologic therapy:

• At least 4 weeks since prior immunotherapy and recovered
• No concurrent immunomodulating agents

Chemotherapy:

• At least 4 weeks since prior chemotherapy (6 weeks for nitrosoureas) and recovered
• No concurrent antineoplastic agents

Endocrine therapy:

• Concurrent corticosteroids for cerebral edema allowed (must be on a stable dose for at least 1 week prior to study entry)

Radiotherapy:

• At least 8 weeks since prior radiotherapy and recovered

Surgery:

• Not specified

Other:

• No prior antineoplaston treatment

Contacts and Locations

Locations

United States, Texas

Burzynski Clinic

Houston, Texas, United States, 77055-6330

Sponsors and Collaborators

Burzynski Research Institute

Investigators

• Principal Investigator: Stanislaw R Burzynski, MD, PhD; Burzynski Research Institute

More Information

Additional Information:

Burzynski Research Institute 

Burzynski Clinic 

• Responsible Party: Burzynski Research Institute
• ClinicalTrials.gov Identifier: NCT00003460
• Other Study ID Numbers: CDR0000066492; BC-BT-12 ( Other Identifier: Burzynski Research Institute, Inc. )
• First Posted: January 27, 2003
• Results First Posted: November 10, 2016
• Last Update Posted: August 24, 2017
• Last Verified: July 2017

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

Keywords provided by Burzynski Research Institute:

Recurrent primitive neuroectodermal tumor

Additional relevant MeSH terms:

Neuroectodermal Tumors; Neuroectodermal Tumors, Primitive; Neoplasms; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms, Nerve Tissue; Neoplasms, Neuroepithelial; Neoplasms, Glandular and Epithelial